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Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

DOI :

10.3791/57530-v

May 11th, 2018

May 11th, 2018

12,743 Views

1Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, 2Muscular Dystrophy Canada Research Chair, Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry

Various antisense oligonucleotides (AONs) have been shown to induce exon inclusion (splice modulation) and rescue SMN expression for spinal muscular atrophy (SMA). Here, we describe a protocol for AON lipotransfection to induce exon inclusion in the SMN2 gene and the evaluation methods to determine the efficacy in SMA patient fibroblasts.

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Exon Inclusion

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